Patent SG-11201806589U-A (SG)
Title: Regulation of gene expression via aptamer-mediated control of self-cleaving ribozymes
Inventor: VOLLES MICHAEL
IPC: C12Q1/68
Filed: 2017-02-02
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization 11M1111101110101011111 HO 11111 01110111001110110011111110111110 1111 OM International Bureau 0.. .... .. ..... ../ (10) International Publication Number (43) International Publication Date ., WO 2017/136608 A3 10 August 2017(10.08.2017) WIPO I PCT (51) International Patent Classification: BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, C12Q 1/68 (2006.01) CO7H 21/02 (2006.01) DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, C07H 21/04 (2006.01) C12N 15/63 (2006.01) HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, C12P 19/34 (2006.01) Cl 2N 15/10 (2006.01) KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, (21) International Application Number: NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, PCT/US2017/016303 RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, (22) International Filing Date: TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, 2 February 2017 (02.02.2017) ZA, ZM, ZW. (25) Filing Language: English (8 4 ) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, (26) Publication Language: English GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, (30) Priority Data: TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, 62/290,187 2 February 2016 (02.02.2016) US TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, 62/290,209 2 February 2016 (02.02.2016) US DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, (71) Applicants: MEIRAGTX UK II LIMITED [GB/GB]; 92 SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, Britannia Walk, London N1 7NQ (GB). MEIRAGTX, GW, KM, ML, MR, NE, SN, TD, TG). LLC [US/US]; 450 East 29th Street, 15th Floor, New York, NY 10016 (US). Declarations under Rule 4.17: (72) Inventors: VOLLES, Michael, J.; 69 Bolton Street, Apt. as to applicant's entitlement to apply for and be granted a —_ patent (Rule 4.1700) = 102, Cambridge, MA 02140 (US). DANOS, Olivier, F.; = 49 Hancock Street, Boston, MA 02114 (US). BOYNE, — as to the applicant's entitlement to claim the priority of the 4.17(iii)) Alex, K.; 198 Van Vorst Street, Apt. 415, Jersey City, NJ earlier application (Rule 07302 (US). ZENNOU, Veronique; 22 Mercer Street, Published: Apt. 2, NJ 07302 Xuecui; 1 Jersey City, (US). GUO, = Bungtown Road, Cold Spring Harbor, NY 11724 (US). — with international search report (Art. 21(3)) Agents: SOMERVILLE, Deborah, A. Fox Roth- — before the expiration of the time limit for amending the (74) et al.; = claims and to be republished in the event of receipt of = schild, LLP, Princeton Pike Corporate Center, 997 Lenox Drive, Building 3, Lawrenceville, NJ 08648-2311 (US). amendments (Rule 48.2(h)) (88) Date of of the international search report: (81) Designated States (unless otherwise indicated, for every publication = kind of national available): AE, AG, AL, AM, 24 August 2017 protection AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, = = = = = = = = M GC © cr) 1-1 (54) Title: REGULATION OF GENE EXPRESSION VIA APTAMER-MEDIATED CONTROL OF SELF-CLEAVING RI- IN BOZYMES 1-1 C (57) : The invention provides polynucleotide constructs for the regulation of gene expression by aptamer-based modulation ei of self-cleaving ribozymes and methods of using the constructs to regulate gene expression in response to the presence or absence of 0 a ligand that binds the aptamer. The invention further provides methods for making and using riboswitches that decrease target gene expression in response to an aptamer ligand as well as riboswitches that increase target gene expression in response to an aptamer ligand.